Paritha Arumugam, Brenna C Carey, Kathryn A Wikenheiser-Brokamp, Jeffrey Krischer, Matthew Wessendarp, Kenjiro Shima, Claudia Chalk, Jennifer Stock, Yan Ma, Diane Black, Michelle Imbrogno, Margaret Collins, Dan Justin Kalenda Yombo, Haripriya Sakthivel, Takuji Suzuki, Carolyn Lutzko, Jose A Cancelas, Michelle Adams, Elizabeth Hoskins, Dawn Lowe-Daniels, Lilith Reeves, Anne Kaiser, Bruce C Trapnell
Pulmonary macrophage transplantation (PMT) is a gene and cell transplantation approach in development as therapy for hereditary pulmonary alveolar proteinosis (hPAP), a surfactant accumulation disorder caused by mutations in CSF2RA/B (and murine homologs). We conducted a toxicology study of PMT of Csf2ra gene-corrected macrophages (mGM-Rα+ Mϕs) or saline-control intervention in Csf2ra KO or wild-type (WT) mice including single ascending dose and repeat ascending dose studies evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics...
June 13, 2024: Molecular Therapy. Methods & Clinical Development